Pipeline News and Updates
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Regulatory Update
The FDA approved elacestrant (Orserdu, Stemline Therapeutics), on 1/27/2023, for the treatment of estrogen receptor–positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy. The FDA approved pirtobrutinib (Jaypirca, Lilly), on 1/27/2023, for the treatment of relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a Bruton's tyrosine kinase inhibitor. The FDA’s Antimicrobial Drugs Advisory Committee voted 14 to 1 to recommend approval of rezafungin for the treatment of candidemia and invasive candidiasis. The FDA accepted the NDA for gepirone or the treatment of major depressive disorder and set a PDUFA date of 6/23/2023. The FDA granted Moderna’s mRNA vaccine, mRNA-1345, a Breakthrough Therapy Designation for the prevention of RSV-associated lower respiratory tract disease in adults aged 60 years or older. The EMA validated the MAA for exagamglogene autotemcel, initiating a full review by CHMP. CHMP recommended that palovarotene to treat fibrodysplasia ossificans progressiva not be approved by the EMA. Announced Research Updates Deciphera announced that in a 453 patient, open-label, Phase III, INTRIGUE trial, treatment with ripretinib did not improve PFS compared to sunitinib in patients with GIST previously treated with imatinib. Peregrine announced that in a 28 patient, Phase II trial, treatment with bavituximab plus pembrolizumab resulted in an ORR of 32.1% in patients with previously untreated advanced hepatocellular carcinoma. Published Research Updates In the 52-week, 159 patient, Phase III, XTEND-1 trial, once weekly treatment with efanesoctocog alfa resulted in an annualized bleeding rate of 0.71 in 133 patients with severe hemophilia A. 26 patents in XTEND-1 received on-demand efanesoctocog alfa and 97% of bleeding episodes resolved after one injection of efanesoctocog alfa. In the 12-week, 140 patient, Phase IIb, ADVISE trial, treatment with etrasimod did not improve the Eczema Area and Severity Index (EASI) compared to placebo in patients with moderate-to-severe atopic dermatitis. In the 24-week, 195 patient, Phase III, MOMENTUM trial, 25% of patients treated with momelotinib achieved a reduction of at least 50% in their Myelofibrosis Symptom Assessment Form (MFSAF) TSS compared to 9% with danazol in patients with myelofibrosis who were symptomatic and anemic and had been previously treated with an FDA-approved JAK inhibitor. We recently added 3 new drugs to the knowledgebase:
The Prescribe Right Pharmaceutical Pipeline Tracker now allows you to track 1,113 pipeline and recently approved drugs supported by 2,952 announced and published study results. Regulatory Update
The FDA approved bexagliflozin (Brenzavvy, TheracosBio), on 1/20/2023, to improve glycemic control in adults with type 2 diabetes mellitus as an adjunct to diet and exercise. The FDA granted granted tecarfarin a Fast Track designation for the prevention of systemic thromboembolism in patients with end-stage renal disease and atrial fibrillation. Finch discontinued the Phase III PRISM4 trial in January 2023, while the company examines the value of its intellectual property estate and other assets. The PRISM4 trial was evaluating CP101 in the prevention of recurrent C. difficile infection. Regulatory Update
Medexus and Medac estimate it will take up to a year to collect and submit all data requested by the FDA for the resubmitted NDA for treosulfan. Announced Research Updates Oramed announced that in the 26-week, 710 patient, Phase III, ORA-D-013-1 trial, oral insulin did not improve HbA1c or fasting plasma glucose compared to placebo in patients with type 2 diabetes that was not controlled by at least one antihyperglycemic agent. Published Research Updates In the 12-week, 153-patient, Phase IIb, D1AMOND trial, treatment with ecopipam reduced the YGTSS-TTS by 3.44 points compared to placebo in patients aged 6 to 17 years with Tourette syndrome. Subscribers received a total of EIGHT study results this week. Contact us today for a subscription. We recently added 25 new drugs to the knowledgebase:
The Prescribe Right Pharmaceutical Pipeline Tracker now allows you to track 1,111 pipeline and recently approved drugs supported by 2,934 announced and published study results Regulatory Update
The FDA approved lecanemab (Leqembi, Eisai & Biogen), on 1/6/2023, too slow the reduction in cognition in Alzheimer’s patients with mild cognitive impairment or mild dementia. Eisai & Biogen set WAC for at lecanemab $26,500 per year. The FDA accepted the BLA for nirsevimab for the prevention of respiratory syncytial virus (RSV) in newborns and infants with an approval decision anticipated in 3Q23. The FDA accepted the BLA for rozanolixizumab for the treatment of generalized myasthenia gravis in patients who are anti-acetycholine receptor or anti-muscle-specific tyrosine kinase antibody positive with an approval decision expected by the end of 2Q23 The FDA accepted the BLA for glofitamab for the treatment of relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy and set a PDUFA date for 7/1/2023. The FDA accepted the NDA for vamorolone for the treatment of Duchenne muscular dystrophy (DMD) and set a PDUFA date for 10/26/2023. The FDA has placed a hold on the launch of vonoprazan until acceptable levels of nitrosamine and met and maintained throughout the shelf-life of the product. Checkpoint Therapeutics submitted a BLA for cosibelimab for the treatment of metastatic cutaneous squamous cell carcinoma. Novan submitted an NDA for berdazimer gel for the treatment of molluscum contagiosum. The FDA granted an Orphan Drug Designation to azeliragon for the treatment of glioblastoma. Regulatory Update
The FDA approved lenacapavir (Sunlenca, Gilead Sciences), on 12/22/2022, for the treatment of HIV-1 infection in heavily treatment-experienced adults with multi-drug resistant HIV-1 infection. Lenacapavir is initiated as a combination of oral tablets with the first subcutaneous injection. After the loading dose, a subcutaneous injection of lenacapavir is given every six months. According to Endpoints News WAC for the first year of lenacapavir will be $42,250 with subsequent years costing $39,000. The FDA approved mosunetuzumab (Lunsumio, Genentech, Roche), on 12/22/2022, for the treatment of relapsed or refractory follicular lymphoma in patients who have received at least two previous systemic therapies. The FDA approved ublituximab (Briumvi, TG Therapeutics), on 12/28/2022, for the treatment of relapsing forms of multiple sclerosis in adults. The FDA delayed a decision on toripalimab, until a site inspection of the manufacturing facility can be completed. Due to COVID-19 related restrictions on travel in China, an inspection had not been competed in December 2022. The FDA rejected palovarotene and requested additional details on clinical trials in December 2022. The FDA accepted the resubmitted BLA for bimekizumab for the treatment of moderate to severe plaque psoriasis with approval expected in 2Q23. The FDA has placed a hold on the Phase III VITESSE trial, evaluating Viaskin Peanut, until DBV makes changes to the study protocol regarding statistical analysis and total number of patients. DBV modified the VITESSE protocol, and the FDA lifted the hold in December 2022. SpringWorks submitted an NDA for nirogacestat for the treatment of adults with desmoid tumors. Published Research Updates In a review of fezolinetant, ICER found evidence to support a health benefit for fezolinetant in the treatment of vasomotor symptoms to be insufficient compared to menopausal hormone therapy. Longer-term safety and efficacy data are needed to determine the place in therapy for fezolinetant. ICER estimated a Health Benefit Price Benchmark for fezolinetant to be $2,000 to $2,500 per year. ICER found evidence to support use of lecanemab to slow the development of mild cognitive impairment in early Alzheimer's disease to be promising but inconclusive with the possibility for harm due to ARIA. ICER estimated a range to meet cost-effectiveness criteria to be $8,500 to $20,600 per year. ICER found evidence to support use of donanemab to slow the development of mild cognitive impairment in early Alzheimer's disease to be insufficient, with only data from a Phase II trial available. There is also the with the possibility for harm due to ARIA. ICER assumed the drug would be as effective as lecanemab to estimate a range to meet cost-effectiveness criteria to be $14,500 and $46,900 per year. An ICER review concluded there was a moderate certainty of a comparable, small, or substantial health benefit with etranacogene dezaparvovec compared to factor IX therapy for the treatment of severe hemophilia B. While etranacogene dezaparvovec offers a clinical benefit, ICER was concerned about the duration of the benefit with long-term data only available through 18 months for 54 patients. There was also concern about potential long-term harms to liver function and hepatocellular carcinoma. ICER estimated a Health Benefit Price Benchmark (HBPB) of $2.93 million to $2.96 million for a single dose of etranacogene dezaparvovec. An ICER review concluded there was a moderate certainty of a comparable, small, or substantial health benefit with valoctocogene roxaparvovec compared to factor VIII prophylaxis for the treatment of severe hemophilia A without inhibitors. When comparing valoctocogene roxaparvovec to emicizumab, ICER found limitations due to the small amount of data available to compare the drugs. They concluded there was low certainty for a net health benefit with valoctocogene roxaparvovec compared to emicizumab. While valoctocogene roxaparvovec offers a clinical benefit, ICER was concerned about the duration of the benefit and potential long-term harms to liver function and hepatocellular carcinoma. ICER estimated a Health Benefit Price Benchmark (HBPB) of $1.96 million for a single dose of valoctocogene roxaparvovec. |
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